EFPIA – Blood cancers don't just give up, so neither do we
How will it help patients?
Current treatment is with aggressive chemotherapy, which has a severe effect on patients’ quality of life, both during and after treatment. Around 50% of patients will relapse, leaving them with few treatment options. CAR-Ts offer the potential of a cure in cancers where options are limited, and may therefore prove life-saving for a select group of patients, increasing life expectancy by nearly 70 years.
How many patients could it help?
There are around 44,000 new cases of blood cancers diagnosed across Europe every year. Globally in 2013, around 600,000 people died from blood cancers. Estimates suggest that if the promise of CAR-Ts is realised, it could amount to 9,400 lives saved and 300,000 life years gained for EU patients diagnosed in 2020.
“CAR-T cell therapies help the body fight back against blood cancer and could replace current chemotherapy treatment. They have the potential to save the lives of thousands of people affected by blood cancer.”
CAR-Ts may offer a one-time solution, displacing the costs of chemotherapy treatments, and reducing expenditure on targeted therapy by between 55 and 100%. The curative promise of CAR-Ts may allow people to live longer, healthier, more productive lives.
Gene therapies offer a potential life change for patients with haemophilia B, and we won’t rest until similar rare diseases can be relieved or even cured.Haemophilia: what is the potential breakthrough? Haemophilia B is a rare, severe, blood disorder that affects patients from birth. A faulty gene means the body cannot produce the protein Factor IX, which is needed for blood clotting. Gene therapy aims to repair the direct cause of genetic disease by introducing DNA into cells to compensate for abnormal or faulty genes. Gene therapies have the potential to relieve or even cure rare diseases where the patient’s quality of life and prognosis are poor, and this may remove the need for surgery or life-long medical interventions.
Around 8,000 people in Europe have haemophilia B, of which 60% live with a severe form of the disease, putting them at risk of bleeds. Gene therapy Phase I/II trials have seen results sufficient to transform the disease into a mild form, while further trials could see a cure. In time, gene therapy approaches could also tackle other diseases like cancer and infections.
“Gene therapies have the potential to relieve or even cure rare diseases, such as haemophilia B, where the patient’s quality of life and prognosis are poor.”
The cost of current treatment is approximately €6 million over a single patient’s lifetime. Gene therapy could decrease emergency hospitalisations by reducing the number of patients suffering serious bleeds. It could also lessen the burden on infusion clinics and cut drug expenditure.
Not all aspects of innovations fit the traditional pharmaceutical delivery pathway, and therefore, it is necessary to upgrade or develop new infrastructure to bring innovative therapies to patients. For example, upfront reimbursement for gene therapy is a challenge because there is currently limited evidence of lasting clinical or economic benefit, leading to uncertainty around its value. In the future, new finance schemes and integrated budgets may help to fund this type of innovation.
Non-small-cell lung carcinoma: what is the potential breakthrough?
Combination therapies use multiple drugs with different modes of action to boost the chance of the patient’s cancer being kept under control, or even cured. Combinations may include immunotherapies, which improve the patient’s immune response, and targeted therapies, which destroy the cancer cells or prevent them from spreading. Combining these therapies promises to deliver superior outcomes when compared with single medicines used in isolation.
How will it help patients?
Chemotherapy remains the standard cure for the majority of patients, which, due to its toxicity, can have a significant impact on quality of life. Combinations therapies may mean that fewer patients have to rely on this form of treatment. These therapies could also extend the lives of patients, increase their quality of life and even offer a cure in patients where a diagnosis would currently be terminal.
How many patients could it help?
With around 400,000 people diagnosed each year in the EU, non-small-cell lung carcinoma (NSCLC) is the third most common form of cancer. Survival rates are poor; only 15% of patients live beyond 5 years after their diagnosis. Through improved NSCLC treatment using combination therapy, 30,000 deaths could be avoided in the EU. Improved survival rates will also help relieve the emotional stress on patients and reduce the care and financial burden on families.
“Combining cancer treatments increases their power, helping people live longer and healthier lives.”
Alzheimer’s disease: what is the potential breakthrough? Existing therapies only treat for the symptoms of Alzheimer’s disease, which is thought to be caused by the build-up of plaques in the brain. New therapies are currently in development for early or mild forms of the disease, where symptoms may not yet be evident. These new treatments have the potential to delay the onset and/or progression of Alzheimer’s by preventing or even reversing the build-up of plaques.
Today, more than 10.5 million patients in Europe live with a form of dementia, of which 60 to 80% present as Alzheimer’s. This number is expected to nearly double over the next 35 years, due to a growing and aging population, reaching over 18 million by 2050. Between 1998 and 2014, terminated Alzheimer’s treatment trials outnumbered approved medicines by 30 to 1 – illustrating the high stakes of investing in Alzheimer’s research. New clinical trial results for Alzheimer’s treatments are expected in 2018.
“Alzheimer’s is one of Europe’s largest public health crises, but new treatments could halt the disease in its tracks – allowing patients to live independent lives, for longer.”
Diabetes: what is the potential breakthrough?
Cell therapy involves injecting or inserting living cells into a patient to treat the cause of their disease. The new cells take over the function of the faulty cells, tackling the disease and restoring health. Established examples of cell therapy include blood transfusions and bone marrow transplants. Cell therapy in type 1 diabetes involves transplanting islet cells from a healthy donor pancreas into the patient, enabling their body to regain control of blood sugar levels.
People with type 1 diabetes have to monitor their blood sugar levels regularly and have multiple injections of insulin every day to keep the levels under control. Uncontrolled and low blood sugar can quickly cause confusion, loss of consciousness, seizures or even death. Cell therapies for type 1 diabetes are expected to provide control of blood sugar without the need to have daily insulin injections, and they may also help to delay the onset of serious long-term health conditions.
Approximately 4 million people live in Europe with type 1 diabetes. Diagnosis of diabetes is growing at a rate of 3 to 5% every year. Around 300,000 cases are in children under the age of 18, of which the average age of diagnosis is 12 year’s old. Cell therapy brings hope through potentially restoring the normal function of the pancreas, restricting the need for insulin therapy to only the most severe cases.
“Cell therapy can help control blood sugar, replacing a lifetime of continuous insulin therapy for patients with type 1 diabetes.”
What is the potential impact on Europe’s healthcare systems?
A lack of sufficient evidence often leads to delayed access for patients and increased costs to manufacturers, which can stifle this kind of innovation. This is particularly true for innovations that have uncertainty associated with clinical trials – for example, when unknown technology is involved. Revising regulatory procedures, providing early and ongoing consultation on clinical trial design and using adaptive licensing are key ways to overcome this challenge.
What might need to change in health service delivery?
Cell therapy represents a rapidly evolving field, with a diverse range of manufacturing and treatment approaches, meaning that conventional non-clinical pharmacology and toxicology studies may not be appropriate. Both manufacturers and stakeholders would need to engage early in the process to build experience with new approaches.
Bacterial infections: what is the potential breakthrough?
Antibacterial resistance is growing, and not enough antibiotics are being produced to tackle the problem. Without action, we will return to the pre-antibiotic era. As many as 300 million people could die prematurely from bacterial infections over the next 35 years, unless new antibacterial treatments are developed. Existing antibiotics act against a wide spectrum of disease-causing and beneficial bacteria. Antibacterial monoclonal antibodies (mAbs) offer more targeted treatment. This specificity means that they can slow the development of antibiotic resistance, and reduce the duration and toxicity of antibiotic treatment.
By targeting conserved pathways and activating the body’s immune system, antibacterial mAbs offer more effective ways of addressing antibiotic resistance. This could extend and save the lives of patients who are infected with drug-resistant strains of bacteria. These antibodies can also improve the patient’s quality of life by reducing toxicity associated with high antibiotic doses.
Every year in the European Union, Iceland and Norway, approximately 25,000 patients die from a serious resistant bacterial infection – most of them in hospital. Antibacterial mAbs have the potential to reduce the number of hospital days and money spent on complications resulting from surgery. This could mean that more lifesaving surgeries could be carried out, and fewer families would be affected by the devastating loss of a family member.
“Antibacterial monoclonal antibodies offer new ways of fighting antibiotic resistance and bacterial infections”
Anti-bacterial resistance results in life-threatening infections. The spread of resistance could mean that the healthcare we take for granted – including routine surgery and cancer treatment – could become a high-risk process.
New antibacterial treatments could reduce the virulence of multi-drug-resistant bacterial infections, and save lives. Developing antibacterial mAbs would also reduce the pressure on developing further antibiotics. Production must be incentivised by adjusting thresholds to take into account the difficulties in providing large patient population studies.